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Latest News: Research

New Phase 2 Study of Repurposed Drug for SMA

06 December 2017 / Posted in: Research

Catalyst Pharmaceuticals have announced that they are to investigate the potential of a drug called Firdapse as a treatment for people with SMA Type 3.

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Update on the Scottish Medicines Consortium's Assessment of Nusinersen

30 November 2017 / Posted in: Research

We have now received a further update and clarification that the Scottish Medicines Consortium will be assessing nusinersen for "pre-symptomatic infantile-onset and later-onset 5q SMA, but not for before birth or Adult Onset SMA".

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The NICE Technology Appraisal Programme: Genetic Alliance UK's Respsonse

22 November 2017 / Posted in: Research

Genetic Alliance voices the Rare Disease Community's concerns about the proposed changes to the NICE Technology Appraisal Programme.

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Biogen SMA Community Update to SMA Europe

17 November 2017 / Posted in: Research

Biogen’s SMA Community Update to SMA Europe covers recent trial results as well as what’s happening in terms of access in Europe and worldwide.

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Updates on nusinersen/Spinraza Trials at the World Muscle Society Meeting

15 November 2017 / Posted in: Research

The 22nd International Annual Congress of the World Muscle Society (WMS) was held October 3rd-7th 2017 in Saint Malo, France. While SMA Support UK did not attend, Biogen have uploaded three posters to their repository that were presented at the meeting.

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Clinicians and Charities Make Further Appeal to NHS England to Widen Scope of SMA Type 1 Nusinersen EAP

30 October 2017 / Posted in: Research

SMA Support UK has backed the call led by Professor Francesco Muntoni not to limit the treatment only to infants with two SMN2 copy numbers.

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APPG Agrees Access To New Medicines For Rare Conditions Isn't Working

26 October 2017 / Posted in: Research

The All Party Parliamentary Group (APPG) on Rare, Genetic and Undiagnosed Conditions has asked Genetic Alliance UK to lead work to propose a solution.

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What Is Happening About Access To Nusinersen?

18 October 2017 / Posted in: Research

We share the frustrations of the SMA Community that there has still been no announcement from NICE about whether it will assess the treatment for continued and wider provision of nusinersen by the NHS. Here is an update of what SMA Support UK is doing.

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AveXis to Imminently Initiate AVXS-101 Pivotal Trial in the US

12 October 2017 / Posted in: Research

The Food and Drug Administration (FDA) has granted permission to AveXis to begin their planned US pivotal trial of their gene therapy drug for SMA, AVXS-101. Avexis say that they are to immediately begin the trial.

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Join MDUK's 'FastTrack' Campaign

11 October 2017 / Posted in: Research

Muscular Dystrophy UK are campaigning for faster access to specialist treatments, including Spinraza for SMA. You can find out more and support the 'FastTrack' Campaign here.

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